Eflornithine (DFMO)
Eflornithine (DFMO) is an old sleeping-sickness drug repurposed as the first oral maintenance therapy for high-risk neuroblastoma, approved in December 2023 to reduce relapse after immunotherapy.
Approved 13 December 2023 (US WorldMeds) for adults and children with high-risk neuroblastoma who achieved at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy. Evidence: single-arm NMTRC003/003B (n=105) compared with an external control from ANBL0032 (EFS HR 0.48, OS HR 0.32), a controversial externally controlled approval. EU applications filed 2025. Two years of twice-daily tablets.
1.DFMO irreversibly inactivates ornithine decarboxylase
- Route
- Oral
- Schedule
- Body-surface-area-based dose twice daily for 2 years (e.g. 768 mg twice daily for BSA >1.5 m2)
- Monitoring
- Hearing (ototoxicity), liver enzymes, counts, growth
- Medicare
- Part D (self-administered)
Oral, self-administered, so it is a Part D drug: covered through a stand-alone Part D plan or Medicare Advantage drug benefit, usually on the specialty tier with 25 to 33% coinsurance until the annual cap ($2,000 in 2025, $2,100 in 2026). Iwilfin for high-risk neuroblastoma maintenance; paediatric, so mostly Medicaid/CHIP and commercial family plans.
- Commercial insurance
- covered with prior authorisation
Covered for FDA-labelled and NCCN-listed uses, but almost always behind prior authorisation confirming diagnosis, biomarker and line of therapy; dispensed through a specialty pharmacy.
Part D out-of-pocket capped at $2,000 (2025) / $2,100 (2026). Medicare patients cannot use manufacturer co-pay cards; charity funds (PAN, HealthWell, CancerCare) and the Extra Help subsidy are the routes.
Sources: Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.
- Notes
- No UK marketing authorisation as of September 2026, so no NICE appraisal. Access only through a clinical trial or a company early access scheme.
- NHS England
- Not routinely funded
Sources: NICE search: eflornithine (DFMO). Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.
Compare all products across the US, EU, UK, Japan, China and Australia →
- 13 Dec 2023ApprovalUS
First oral maintenance therapy; externally controlled evidence source
- 2025Filing / dealEU
MAA submitted to EMA, UK, Australia, Switzerland source
Approvals
| Region | Year | Indication |
|---|---|---|
| US | 2023 | Maintenance to reduce relapse risk in high-risk neuroblastoma after ≥PR to prior therapy including anti-GD2 |
| Adverse event | Any grade | Grade 3+ |
|---|---|---|
| Hearing loss | 27% | — |
| Otitis media | 27% | — |
| Neutropenia | 24% | — |
| Elevated ALT | 33% | — |
NMTRC003/003B; largely pre-existing from platinum. Events listed without rates were not read from a primary source; see the label. Blank cells mean the figure was not sourced, not that it is zero.
Trials
topRecruiting now (live from ClinicalTrials.gov)
Country and place are remembered in this browser only. A postcode is sent to OpenStreetMap's Nominatim service to find coordinates when you press the button; nothing else leaves your device.
Landmark trials in OnCo
Latest papers
topQuery for this drug: (TITLE:"Eflornithine" OR ABSTRACT:"Eflornithine" OR TITLE:"DFMO" OR ABSTRACT:"DFMO" OR TITLE:"Iwilfin" OR ABSTRACT:"Iwilfin") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Eflornithine (DFMO), not a curated reading list.
Pages like this
not linked directly; found by shared links- PersonSusan L. Cohn
- IdeaOne global paediatric cancer development plan instead of separate FDA and EMA plans
Shares Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
- TrialNaxitamab Study 201
Shares Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
- TermINRG staging and risk groups
Shares Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
- IdeaGuaranteed-quality childhood cancer medicines free of charge in 50 countries
Shares Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
- PersonAlexandra "Alex" Scott
Shares Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
- IdeaA paediatric palliative care team in every childhood cancer unit
Shares Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
- IdeaMake paediatric combination studies part of every relevant adult cancer drug approval
Shares Neuroblastoma (paediatric), Rare and paediatric cancers without markets.